Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.

New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial

"The advancement of neladalkib from first clinical trial initiation to NDA submission in less than four years represents a remarkable pace in oncology drug development, underscoring the vigor and urgency our team brought to this program and our deep commitment to the ALK-positive NSCLC community," said Darlene Noci, A.L.M., Chief Development Officer at Nuvalent. "We would like to extend our sincere gratitude to the patients, families and investigators who have made this progress possible, and are committed to working closely with the FDA throughout the NDA review process toward our goal of bringing neladalkib to patients as quickly as possible."

The application is based on data in TKI pre-treated patients with advanced ALK-positive NSCLC treated with neladalkib in the global, registration-directed ALKOVE-1 Phase 1/2 clinical trial. In this population, neladalkib demonstrated encouraging overall activity, including intracranial responses, the ability to address key drivers of disease progression, and a generally well-tolerated safety profile consistent with its ALK-selective, TRK-sparing design. The company plans to share detailed results at a future medical meeting.

Neladalkib has received breakthrough therapy designation from the FDA for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About Neladalkib

Neladalkib is an investigational, brain-penetrant, ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About the ALKOVE-1 Phase 1/2 Clinical Trial

The ALKOVE-1 trial (NCT05384626) is a first-in-human Phase 1/2 clinical trial for patients with advanced ALK-positive NSCLC and other solid tumors. The completed Phase 1 portion enrolled ALK-positive NSCLC patients who previously received at least one ALK TKI, or patients with other ALK-positive solid tumors who had been previously treated or for whom no satisfactory standard of care exists. The Phase 1 portion of the trial was designed to evaluate the overall safety and tolerability of neladalkib, with additional objectives including determination of the recommended Phase 2 dose (RP2D), characterization of the pharmacokinetic profile, and evaluation of preliminary anti-tumor activity. The global, single arm, open label Phase 2 portion is designed with registrational intent for TKI pre-treated patients with advanced ALK-positive NSCLC. Global enrollment in ALKOVE-1 remains ongoing for adult and adolescent patients with ALK-positive solid tumors outside of NSCLC, and adolescent patients with ALK-positive NSCLC.

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Some Improvement Seen in Cognitive Function With Exercise, Ibuprofen in Cancer

In continution of my update on Ibuprofen 

For adults with cancer receiving chemotherapy, an exercise intervention and low-dose ibuprofen improved some domains of cognitive function, according to a study published online April 20 in Cancer.

Michelle C. Janelsins, Ph.D., M.P.H., from the University of Rochester in New York, and colleagues randomly assigned 86 adult participants with cancer receiving chemotherapy and reporting cognitive problems to one of four study arms for six weeks: Exercise for Cancer Patients (EXCAP)-ibuprofen, EXCAP-placebo, ibuprofen only, and placebo only. EXCAP is a home-based, low- to moderate-intensity progressive walking and resistance exercise prescription.

The researchers found that compared with the placebo group, participants in the EXCAP-placebo group had significantly better attention performance on the Trail Making Test (–21.57 seconds). Greater improvements were seen for the ibuprofen-only group versus the placebo group (difference of –11.27 seconds). Improvements on the Functional Assessment of Cancer Therapy-Cognitive Function (FACT-Cog) "comments from others" subscale were exhibited by participants in both the EXCAP-ibuprofen and EXCAP-placebo groups compared with the placebo group. Participants in the EXCAP-placebo group had a significant improvement on Rapid Visual Processing mean latency compared with the placebo group; those receiving ibuprofen had an improvement compared with those receiving placebo, which was attributed to a trend after adjustment for reading score. Compared with those not receiving ibuprofen, the ibuprofen group performed less well on the Hopkins Verbal Learning Test‐Revised delayed.

"We are encouraged by the findings of this trial that suggest possible benefits of both interventions for some cognitive domains. Clearly, we saw a more pronounced effect with exercise, which is notable considering the multiple health benefits of exercise for cancer survivors," Janelsins said in a statement.

https://en.wikipedia.org/wiki/Ibuprofen

Some Improvement Seen in Cognitive Function With Exercise, Ibuprofen in Cancer - Drugs.com MedNews

Combining Cannabinoid With Opioid Does Not Relieve Pain in Knee Osteoarthritis

For patients with knee osteoarthritis, combining a cannabinoid medication with an opioid does not provide additional pain relief, according to a study published in the May issue of Anesthesiology.

Katrina R. Hamilton, Ph.D., from Johns Hopkins University School of Medicine in Baltimore, and colleagues examined the acute analgesic and drug effects of synthetic delta-9-tetrahydrocannabinol and hydromorphone, alone and in combination, in 21 individuals with knee osteoarthritis (mean age, 63.4 years). Participants received oral combinations of placebo, hydromorphone (2 mg), and dronabinol (10 mg).

The researchers found greater pressure pain threshold analgesia for hydromorphone than dronabinol and greater capsaicin and noncapsaicin sensitized mechanical temporal summation analgesia versus placebo. No significant drug-related differences were seen for clinical pain severity, thermal threshold or tolerance, temporal summation, cold pressor, conditioned pain modulation, capsaicin-induced thermal threshold, central sensitization, general pain sensitivity, or physical functioning (two-minute walking distance, Timed Up and Go, and total stair climb time). Compared with all conditions, hydromorphone impaired working memory accuracy and produced greater good effects than placebo in terms of secondary outcomes. Working memory reaction time was impaired with hydromorphone + dronabinol, which produced greater high ratings than placebo, greater drug effects than placebo + hydromorphone, and higher nausea than hydromorphone. Greater high ratings were seen for dronabinol than hydromorphone.

"Some patients believe combining cannabis with opioids can help with pain, and clinicians may recommend or prescribe it in states where cannabis is legal," Hamilton said in a statement. "Our study suggests that isn't the case and patients may experience more side effects when the drugs are combined."

https://en.wikipedia.org/wiki/Cannabinoid

https://en.wikipedia.org/wiki/Opioid

Combining Cannabinoid With Opioid Does Not Relieve Pain in Knee Osteoarthritis - Drugs.com MedNews

FDA Approves Idvynso (doravirine and islatravir) for the Treatment of HIV-1 Infection in Adults

In continuation of my update on Doravirine

Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced the U.S. Food and Drug Administration (FDA) approval of  Idvynso, a new, two-drug single-tablet regimen of 100 mg doravirine and 0.25 mg islatravir, for the treatment of HIV-1 infection in adults to replace the current antiretroviral regimen in those who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) on a stable antiretroviral regimen with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine. Idvynso is contraindicated when co-administered with drugs that are strong cytochrome P450 (CYP)3A enzyme inducers and lamivudine (3TC) or emtricitabine (FTC). Co-administration with these drugs may decrease the effectiveness of Idvynso. See additional selected safety information on the following pages. Idvynso (pronounced ihd-VIHN-soh) will be available in pharmacies after May 11.

                                                                Islatravir

Doravirine

• Idvynso is approved for adults with virologically suppressed HIV-1 with no history of virologic treatment failure and no known substitutions associated with resistance to doravirine
• Idvynso is the first and only non-INSTI, tenofovir-free, once-daily, complete two-drug regimen to demonstrate non-inferior efficacy in a head-to-head Phase 3 trial versus three-drug regimen Biktarvyi (BIC/FTC/TAF)

“Advances in HIV treatment mean more people living with HIV are living longer — a remarkable achievement,” said Carl Baloney, Jr., president and chief executive officer of AIDS United. “People aging with HIV face additional health challenges, including managing multiple chronic conditions and medications at the same time. It is essential that management of HIV considers these factors in addition to virologic suppression when choosing an HIV treatment regimen.”

“Idvynso combines islatravir, a next-generation NRTI with multiple mechanisms of action, including translocation inhibition, with doravirine, an NNRTI with an established efficacy and safety profile. As the only two-drug, non-INSTI, tenofovir-free regimen, Idvynso expands therapeutic diversity beyond the currently available oral treatment options,” said Dr. Eliav Barr, senior vice president and chief medical officer, Merck Research Laboratories. “As the health needs of adults living with HIV change over time, Idvynso gives clinicians a new choice for HIV treatment. This approval marks an important new chapter in Merck’s long-standing commitment to research and discovery for people living with HIV.”

Idvynso is a complete regimen; co-administration with other antiretroviral medications for treatment of HIV-1 infection is not recommended. Severe skin reactions, including Stevens-Johnson syndrome/toxic epidermal necrolysis, have been reported with doravirine-containing regimens. Drug Rash with Eosinophilia and Systemic Symptoms was reported with Idvynso. Concomitant use of Idvynso and certain other drugs may result in known or potentially significant drug interactions, some of which may lead to loss of therapeutic effect of Idvynso and possible development of resistance, or possible clinically significant adverse reactions from greater exposures of a component of Idvynso. See additional selected safety information on the following pages.

“Idvynso is the first non-INSTI, tenofovir-free, two-drug regimen to demonstrate non-inferior efficacy to standard oral antiretroviral regimens, including Biktarvy. This makes Idvynso a potential alternative for people with virologically suppressed HIV who may need to switch their treatment,” said Dr. Amy Colson, director of research at Community Resource Initiative, Boston, Massachusetts.

Phase 3 studies supporting approval of Idvynso

The efficacy and safety of Idvynso is supported by Week 48 data from two randomized, active-controlled, non-inferiority trials [Trial 052 (NCT05630755) and Trial 051 (NCT05631093)] in virologically-suppressed (HIV-1 RNA less than 50 copies per mL) adults living with HIV. Participants must have been stably suppressed on their baseline regimen for at least 3 months prior to trial entry and had no history of treatment failure. Across the two trials, a total of 708 participants received once-daily Idvynso; of these, 81 (11%) participants were aged 65 years and older, including 10 (1%) aged 75 years and older.

In the double-blind Trial 052, participants were switched from Biktarvy [bictegravir/emtricitabine/tenofovir alafenamide (BIC/FTC/TAF)] to Idvynso. A total of 513 participants were randomized (2:1) and were switched to once-daily Idvynso (n=342) or remained on BIC/FTC/TAF (n=171). At baseline, participants had a mean age of 48 years (range: 19 to 77), 21% of participants were female, 61% were White, 31% were Black/African American, and 6% were Asian. A total of 23% identified as Hispanic/Latino.

In the open-label Trial 051, participants were switched from an oral ART (antiretroviral therapy) regimen to Idvynso. A total of 551 participants were randomized (2:1) and were switched to once-daily Idvynso (n=366) or remained on their baseline ART (bART) (n=185). Randomization was stratified by bART. At baseline, participants had a mean age of 50 years (range: 18 to 83), 40% of participants were female, 39% were White, 45% were Black/African American, and 5% were Asian. A total of 15% identified as Hispanic/Latino. At enrollment, 64% of the participants were receiving integrase strand transfer inhibitor (INSTI)-based regimens, 5% protease inhibitor (PI)-based regimens (including combinations with INSTI), and 30% were receiving other regimens.

https://en.wikipedia.org/wiki/Islatravir

https://en.wikipedia.org/wiki/Doravirine

FDA Approves Idvynso (doravirine and islatravir) for the Treatment of HIV-1 Infection in Adults

Injectable peptides are the new anti‑aging trend. But what evidence do we have they're safe for humans?

Injectable peptides are the new anti-aging trend sweeping the beauty industry. These compounds are promoted on social media as tools for skin repair, collagen production and "cellular rejuvenation." They are widely available online from overseas sellers, despite many peptides being unregulated in Australia.

But what's in them? And are they safe?

Earlier this year, three people in the United States were fined thousands of dollars for their role in providing peptide injections, at an anti-aging festival in Las Vegas, to two women who later became critically ill. The pharmacy board was unable to determine why they got sick, and what precisely the serums contained.

Our work with colleagues at Steroid QNECT, a hotline where people can seek confidential advice about enhancement drugs, shows that people are already injecting peptides in Australia.

But regulation is not keeping up. And there are still major gaps in the evidence about whether peptides' anti-aging claims stack up, and whether they are safe for humans.

What are injectable peptides, and why are they trending?

Peptides are short chains of amino acids—the building blocks of proteins. They act as chemical messengers in the body and play a key role in many processes. These include helping repair skin and calming inflammation.

The body naturally produces peptides. Synthetic peptides are manufactured to mimic or enhance these natural functions.

Certain peptides have clear medical uses. For example, glucagon-like peptide-1 (GLP-1) drugs, such as semaglutide, are approved for diabetes and weight management—sold as Ozempic and Wegovy. This is based on strong clinical trial evidence that they are effective.

But a growing number of peptides are being marketed for cosmetic and anti-aging purposes, without approval from Australia's therapeutic goods regulator.

Peptides such as GHK-Cu, BPC-157 and TB-500 are sold online with claims they can enhance collagen production, accelerate skin repair, reduce wrinkles, and even reverse aspects of biological aging.

TB 500

BPC-157

GHK-Cu

https://en.wikipedia.org/wiki/TB-500

https://en.wikipedia.org/wiki/BPC-157

https://en.wikipedia.org/wiki/Copper_peptide_GHK-Cu

Experimental drug cuts Parkinson's-linked protein up to 60% in early trial

An experimental drug designed to silence a gene strongly linked to Parkinson's disease has shown encouraging effects in a first-in-human clinical trial, according to a study published in Nature Medicine. The drug, known as   BIIB094 (ION859), an antisense oligonucleotide targets LRRK2, the most common genetic contributor to Parkinson's disease; variants in LRRK2 are known to increase the risk of developing the neurodegenerative disorder, which affects nearly 10 million people worldwide

Scientists have long believed that lowering the activity of the LRRK2 protein could help slow or modify the disease, but turning that idea into a viable therapy has remained a challenge, said co-author of the study Danielle Larson, MD, '15, '18 GME, assistant professor in the Ken & Ruth Davee Department of Neurology's Division of Movement Disorders.

"This was a multicenter clinical trial looking at an antisense oligonucleotide therapy for LRRK2-specific Parkinson's disease," Larson said. "The main goal was to examine the safety of delivering this therapy to patients, with the hope that if it proved safe, future studies could evaluate whether it might slow disease progression."

The study tested whether BIIB094 could safely reduce LRRK2 levels in people with Parkinson's disease. The results suggest it can be done without serious safety concerns, Larson said.

In the randomized, placebo-controlled trial, 82 participants with Parkinson's disease were enrolled across two study segments. In the first, 40 participants received a single dose of BIIB094 or placebo. In the second, 42 participants received four doses of the drug or placebo, administered every four weeks. The therapy was delivered intrathecally: directly into the cerebrospinal fluid through a lumbar puncture.

Participants in the second section were stratified based on whether they carried a known LRRK2 genetic variant.

Across both parts of the trial, the treatment was generally well tolerated. While adverse events were common, they were mostly mild to moderate and did not limit dosing. No serious adverse events related to BIIB094 were reported, according to the study.

Beyond safety, the study provided strong evidence that BIIB094 hit its intended biological target. Analysis of cerebrospinal fluid showed that levels of LRRK2 protein fell by as much as 59% in treated participants.

These reductions were observed regardless of whether patients carried a known LRRK2 mutation, suggesting the therapy could potentially affect a broader group of Parkinson's patients, not just those with a genetic diagnosis, Larson said.

"The antisense oligonucleotide was designed specifically to reduce LRRK2 expression," Larson said. "Because overactivity of this protein kinase is thought to be part of the problem in Parkinson's disease, reducing LRRK2 levels could be protective and potentially modify the disease."

The new findings hint at a possible mechanism by which LRRK2-targeting therapies could influence the underlying disease process, rather than simply addressing symptoms, Larson said.

However, Larson cautioned that the trial was not designed to assess clinical benefits such as changes in movement, cognition, or disease progression. Larger and longer studies will be needed to determine whether reducing LRRK2 levels translates into meaningful improvements for patients.

"The next step would be a phase 2 study with a larger group of patients," Larson said. "Instead of focusing only on safety, those trials would look at efficacy, whether the therapy can slow disease progression using motor assessments and standard Parkinson's rating scales."

The findings mark an important step toward targeted treatments that address the root biology of Parkinson's disease and underscore the potential of BIIB094 and other precision-medicine approaches, Larson said.

"This is one of the first antisense oligonucleotide, or ASO, therapies in Parkinson's disease to have safety and tolerability data," Larson said. "It really paves the way for other ASO-based treatments to be developed, potentially targeting different biological pathways."

https://www.nature.com/articles/s41591-026-04262-4

Experimental drug cuts Parkinson's-linked protein up to 60% in early trial

Immunome Announces Submission of New Drug Application to U.S. FDA for Varegacestat for the Treatment of Adults with Desmoid Tumors

I

Immunome, Inc. (Nasdaq: IMNM), a biotechnology company committed to developing first-in-class and best-in-class targeted cancer therapies,  announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for varegacestat, an investigational, oral, once-daily gamma secretase inhibitor (GSI), for the treatment of adults with desmoid tumors.

• New Drug Application (NDA) supported by positive Phase 3 RINGSIDE results, including significant improvement in progression-free survival vs. placebo (hazard ratio = 0.16, p<0.0001)
• Trial also met all key secondary endpoints, with varegacestat delivering an objective response rate of 56%
• Detailed Phase 3 RINGSIDE data selected for oral presentation at the 2026 ASCO Annual Meeting

“The varegacestat NDA submission marks an important milestone for Immunome. It reflects the strength of the RINGSIDE dataset and the commitment of the team advancing this program,” said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Immunome. “We believe varegacestat has the potential to provide adults with progressing desmoid tumors with a meaningful new oral treatment option, and we are grateful to the patients, families, investigators and study site teams whose participation made this submission possible.”

The NDA is supported by positive results from the global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial of varegacestat in patients with progressing desmoid tumors.

The trial met its primary endpoint of improving progression-free survival, demonstrating a statistically significant and clinically meaningful improvement vs. placebo, with an 84% reduction in the risk of disease progression or death (hazard ratio (HR) = 0.16, 95% CI: 0.071, 0.375; p<0.0001). The confirmed objective response rate (ORR) based on RECIST v1.1 was 56% with varegacestat vs. 9% with placebo (p<0.0001), as assessed by blinded independent central review.

In an exploratory analysis, varegacestat demonstrated a median best change in tumor volume of -83% vs. +11% with placebo, as assessed by blinded independent central review. In addition, the trial met all key secondary endpoints, with varegacestat achieving statistically significant improvements vs. placebo in landmark tumor volume reduction and worst pain intensity.

Varegacestat was generally well tolerated, with a manageable safety profile consistent with the GSI class. The most common adverse events for participants in the treatment arm were diarrhea (82%), fatigue (44%), rash (43%), nausea (35%) and cough (34%). Most events were grade 1 or 2.

Immunome previously announced that data from RINGSIDE has been selected for presentation in an oral abstract session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 29-June 2, 2026 in Chicago.

About the RINGSIDE Trial

The global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial (NCT04871282) evaluated the efficacy and safety of varegacestat in patients with progressing desmoid tumors. A total of 156 patients were randomized to receive varegacestat 1.2 mg daily or placebo until disease progression or death, representing the largest randomized study in this population. The primary endpoint of the trial was progression-free survival as assessed by blinded independent central review. Statistically controlled secondary endpoints were confirmed ORR using RECIST v1.1 and change in tumor volume at week 24, both determined by blinded independent central review, as well as change in pain intensity at week 12 as determined using a patient reported outcome instrument. Additional secondary endpoints included duration of response, best reduction in tumor volume, patient-reported outcomes, and safety and tolerability. RINGSIDE includes an open-label extension phase, which is ongoing.

About Desmoid Tumors

Desmoid tumors (also known as aggressive fibromatosis or desmoid-type fibromatosis) are aggressive non-metastatic soft tissue tumors that are prone to recurrence. Approximately 1,000-1,650 people are diagnosed with desmoid tumors each year in the United States, and there are approximately 10,000-11,000 actively managed patients. Those affected face debilitating pain, deformity and, in some cases, life-threatening organ damage. The chronic pain and physical limitations associated with desmoid tumors lead to a high clinical burden and impaired quality of life. Although desmoid tumors are not considered cancerous, they often require systemic treatment to prevent permanent disability and alleviate disease burden.

About Varegacestat

Varegacestat (formerly AL102) is an investigational, oral, once-daily gamma secretase inhibitor. In December 2025, Immunome reported positive topline results for the Phase 3 RINGSIDE trial of varegacestat in adults with progressing desmoid tumors. Data from RINGSIDE have been selected for oral presentation at the 2026 ASCO Annual Meeting.

https://www.medkoo.com/products/7653

mmunome Announces Submission of New Drug Application to U.S. FDA for Varegacestat for the Treatment of Adults with Desmoid Tumors

FDA Accepts New Drug Application for Zipalertinib for the Treatment of Non-Small Cell Lung Cancer

Taiho Oncology, Inc., Taiho Pharmaceutical Co., Ltd., and Cullinan Therapeutics, Inc.(Nasdaq: CGEM) announced  the U.S. Food and Drug Administration (FDA)  acceptance of  New Drug Application (NDA) for zipalertinib for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion (ex20ins) mutations whose disease has progressed on or after platinum-based chemotherapy, with or without amivantamab. The Prescription Drug User Fee Act (PDUFA) target action date is February 27, 2027.

• NDA submission based on the Phase 2b REZILIENT1 clinical trial, which demonstrated clinically meaningful and durable responses in patients with relapsed EGFR exon 20 insertion–mutated NSCLC
• Prescription Drug User Fee Act (PDUFA) target action date is February 27, 2027

The NDA is supported by data from the Phase 2b part of the REZILIENT1 clinical trial of zipalertinib monotherapy in patients with NSCLC harboring EGFR ex20ins mutations who have received prior therapy. The study met its primary endpoint of objective response rate. Study results from REZILIENT1 were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and simultaneously published in the Journal of Clinical Oncology.

“Zipalertinib was discovered at Taiho Pharmaceutical Co., Ltd., and has been developed with a focus on addressing the unmet needs of patients with EGFR exon 20 insertion-mutated non-small cell lung cancer,” said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. “The FDA’s acceptance of the NDA for zipalertinib is an important milestone for this program, and we look forward to working with FDA during the review process.”

“Zipalertinib is a compound created using Taiho Pharmaceutical’s proprietary drug discovery and development technologies, Cysteinomix, with the aim of delivering a new treatment option to address high unmet medical needs,” said Takeshi Sagara, PhD, Executive Director, Board Member, Medical Affairs, Translational Development, Clinical Development, Discovery and Preclinical Research at Taiho Pharmaceutical. “The FDA’s acceptance of the NDA represents an important milestone, reflecting the scientific and clinical data accumulated to date. We will continue to work closely with Taiho Oncology, Cullinan Therapeutics and the FDA throughout the review process, with the shared goal of ultimately delivering a new treatment option to patients with non-small cell lung cancer EGFR exon 20 insertion mutations.”

“FDA acceptance of the zipalertinib NDA is an important step toward making zipalertinib available for people living with non-small cell lung cancer with EGFR exon 20 insertion mutations, who continue to face limited treatment options,” said Jeffrey Jones, MD, MBA, Chief Medical Officer, Cullinan Therapeutics. “We are deeply grateful to the patients and families who have participated in the REZILIENT program, and to the investigators, study teams, and advocates whose collaboration made achievement of this milestone possible. We believe zipalertinib has the potential to help address a significant unmet need, and we look forward to working with our partners at Taiho with the goal of bringing zipalertinib to patients waiting for new treatment options.”

Summary of Primary Study Results:

• Zipalertinib demonstrated clinically meaningful efficacy in the primary efficacy population (n=176), including 51 patients who had received prior amivantamab.
• The confirmed objective response rate (ORR) was 35%. Median duration of response (mDOR) was 8.8 months
• In patients treated after prior platinum-based chemotherapy only (n=125), ORR was 40% with a mDOR of 8.8 months.
• In exploratory subgroup analyses:
  • Patients who had received prior amivantamab without other ex20ins-targeted therapy (n=30) showed a confirmed ORR of 30% and mDOR of 14.7 months.
  • Patients with brain metastases (n=68) showed a confirmed ORR of 31% and a mDOR of 8.3 months.
• The safety profile of zipalertinib was manageable and consistent with previously reported data.¹ The most common treatment-emergent adverse events were paronychia, rash, anemia, dermatitis acneiform, diarrhea, dry skin, nausea and stomatitis. Most treatment-emergent adverse events were grade 1 or 2 per NCI-Common Terminology Criteria for Adverse Events (CTCAE v5.0).

Zipalertinib is an oral EGFR tyrosine kinase inhibitor. Zipalertinib received Breakthrough Therapy Designation in 2021 for the treatment of patients with locally advanced or metastatic NSCLC harboring EGFR ex20ins mutations who have previously received platinum‑based systemic chemotherapy.

https://en.wikipedia.org/wiki/Zipalertinib

U.S. Food and Drug Administration Accepts New Drug Application for Zipalertinib for the Treatment of Locally Advanced or Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

New drug combination doubles down on Alzheimer's treatments

A new study has found that combining the current medications for Alzheimer's disease with small molecules derived from micronutrients found in grapes, berries, peanuts and turmeric is a safer and more effective way to treat the disease. The work is published in the journal ACS Chemical Neuroscience.

Individuals with Alzheimer's have a buildup of toxic amyloid proteins in the brain. Researchers from the School of Pharmacy at the University of Waterloo combined amyloid-destroying small molecules with anti-amyloid antibodies that are already used in Alzheimer's treatment. They found that it neutralized the clumping of proteins that accumulate in the brain, leading to better outcomes.

Alzheimer's is the major cause of dementia. Dementia affects nearly 750,000 people in Canada, with a million cases expected by 2030. Alzheimer's has no cure and current medications only relieve a patient's symptoms. Anti-amyloid antibody therapies on their own can slow the disease, but they also come with risks that can be fatal, including brain swelling and bleeding.

"We already know the small molecules resveratrol or curcumin, which are found in some common foods, block the buildup of amyloid," said Dr. Praveen Nekkar Rao, a professor in the School of Pharmacy at Waterloo. "What's new and exciting is our combination of these molecules with the anti-amyloid antibodies. This approach could allow clinicians to use lower doses of antibodies, potentially reducing the risk of serious treatment-related side effects."

Since there are few effective treatments for Alzheimer's, researchers at Waterloo studied whether using two treatments together could work better than using just one. They chose resveratrol and curcumin because they are natural compounds known to reduce amyloid buildup and inflammation.

"I was inspired by chemotherapy, which involves taking multiple medications for effective treatment," Nekkar Rao said. "Alzheimer's is a complex disease, but there are very few combination therapy approaches. Our results show that the way forward is definitely combination therapy."

The researchers emphasize that the study does not suggest that people should start consuming resveratrol or curcumin to prevent or treat dementia. You would have to consume an unsafe amount in order to reach the brain. The next phase of the research will focus on designing next-generation drugs that can reach the brain more effectively, interact favorably with amyloids and pair seamlessly with antibody treatments.

https://pubs.acs.org/doi/10.1021/acschemneuro.5c00760

New drug combination doubles down on Alzheimer's treatments

U.S. FDA Grants Full Approval of Kite’s Tecartus for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma

The U.S. Food and Drug Administration (FDA) has granted traditional (full) approval to Kite’s CAR T-cell therapy, Tecartus (brexucabtagene autoleucel), for adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL). The full approval now includes efficacy, safety and pharmacokinetic data from Cohort 3 of the ZUMA-2 study in patients who are R/R after one or more lines of therapy and who are Bruton tyrosine kinase inhibitor (BTKi)-naïve.

Today’s action converts the R/R MCL indication to full approval based on the totality of evidence from ZUMA-2, including confirmatory data from Cohort 3, which demonstrated high response rates, durable efficacy outcomes and a manageable safety profile consistent with prior experience. This milestone fulfills Kite’s post-marketing requirement for verification and description of clinical benefit in a confirmatory trial under the FDA’s Accelerated Approval pathway for Tecartus in R/R MCL.

“The full approval of brexu-cel in relapsed or refractory mantle cell lymphoma, along with the inclusion of Cohort 3 data in the label, reinforces our confidence in the overall profile of brexu-cel,” said Michael Wang, M.D., ZUMA-2 Lead Investigator and Professor, Department of Lymphoma and Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center. “The Cohort 3 results showed high response rates, including deep remissions, in patients who were Bruton tyrosine kinase inhibitor-naïve, with a manageable safety profile consistent with prior experience. These data provide important information to help guide treatment decisions in the relapsed or refractory setting for appropriate patients.”

MCL is a rare form of non-Hodgkin lymphoma (NHL) that arises from cells originating in the “mantle zone” of the lymph node and predominantly affects men over the age of 60. Approximately 33,000 people worldwide are diagnosed with MCL each year. MCL is highly aggressive following relapse, with many patients’ disease progressing following therapy.

“The full approval of Tecartus for adults with relapsed or refractory mantle cell lymphoma reflects meaningful progress,” said Gallia Levy, MD, PhD, Senior Vice President and Global Head of Development, Kite. “For those whose cancer has returned, the data support Tecartus as a second-line treatment option with the potential to deliver long-term remission.”

Data Supporting Full Approval of Tecartus in R/R MCL

ZUMA-2 is a single-arm, open-label, multicenter study evaluating Tecartus in adult patients with R/R MCL. Cohorts 1 and 2 evaluated Tecartus in patients who had previously received up to five lines of therapy including anthracycline- or bendamustine-containing chemotherapy, an anti-CD20 antibody and a BTKi. Cohort 3 evaluated Tecartus in patients who had received up to five prior lines of therapy and were BTKi-naïve. A total of 82 patients were treated in Cohorts 1 and 2, and 86 patients were treated in Cohort 3. The primary endpoint across the study was objective response rate (ORR) per the Lugano Classification (2014), as assessed by an Independent Radiologic Review Committee.

U.S. FDA Grants Full Approval of Kite’s Tecartus for Adult Patients with Relapsed or Refractory Mantle Cell Lymphoma

FDA Approves Revtorpyk (gedatolisib) for the Treatment of HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer

Celcuity Inc. (Nasdaq: CELC), a biotechnology company focused on developing and commercializing targeted therapies for multiple solid tumor indications,   announced  the U.S. Food and Drug Administration (“FDA”) approval of  Revtorpyk (gedatolisib) for the treatment of patients with hormone receptor positive (“HR+”), human epidermal growth factor receptor 2 negative (“HER2-”), locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting. Revtorpyk is the only inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2 to receive FDA approval.

• Revtorpyk is the first and only FDA-approved therapy that inhibits all class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2
• In the Phase 3 VIKTORIA-1 trial, Revtorpyk combined with palbociclib and fulvestrant and Revtorpyk combined with fulvestrant reduced the risk of disease progression or death by 76% and 67%, respectively, compared to fulvestrant among patients with PIK3CA wild-type advanced or metastatic breast cancer

“The PI3K/AKT/mTOR, or PAM, pathway is one of the most important targets in cancer, but comprehensively inhibiting it has stymied researchers and drug developers for nearly two decades,” said Brian Sullivan, CEO and co-founder of Celcuity. “Revtorpyk addressed this 20-year challenge by becoming the first pan-PI3K, mTORC1/2 inhibitor approved by the FDA. We are thankful for the opportunity to make this important new therapy available to patients with HR+/HER2- locally advanced or metastatic breast cancer.”

HR+/HER2- breast cancer is the most common subtype of breast cancer, accounting for approximately 70% of all breast cancers.1 Among this breast cancer subtype, approximately 60% have PIK3CA wild-type disease.2

“For patients with HR+/HER2- locally advanced or metastatic breast cancer, there is an urgent need for new treatment options that can meaningfully increase the likelihood of survival without disease progression or death,” said Sara Hurvitz, MD, Senior Vice President, Clinical Research Division, Fred Hutchinson Cancer Center, Smith Family Endowed Chair in Women’s Health and Professor and Head, Division of Hematology and Oncology, University of Washington, School of Medicine and co-principal investigator for the VIKTORIA-1 trial. “With the approval of Revtorpyk, oncologists now have an effective new treatment option for these patients.”

The approval of Revtorpyk is based on positive clinical results from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial, an open-label, global, randomized clinical trial evaluating the efficacy and safety of Revtorpyk plus fulvestrant, with or without palbociclib, for the treatment of patients with locally advanced or metastatic HR+/HER2- breast cancer following progression on or after CDK4/6 therapy and an aromatase inhibitor. In the VIKTORIA-1 trial, median progression free survival (“PFS”) with the Revtorpyk triplet (Revtorpyk plus palbociclib and fulvestrant) was 9.3 months versus 2.0 months with fulvestrant, an incremental improvement of 7.3 months (HR=0.24; 95% CI: 0.17-0.35; p<0.0001). The objective response rate (“ORR”) of the Revtorpyk triplet was 32% compared to 1% with fulvestrant and the median duration of response (“DOR”) was 17.5 months. For the Revtorpyk doublet (Revtorpyk plus fulvestrant), the median PFS was 7.4 months versus 2.0 months with fulvestrant, an incremental improvement of 5.4 months (HR=0.33; 95% CI: 0.24-0.48; p<0.0001). The ORR of the Revtorpyk doublet was 28% and the median DOR was 12.0 months. The median DOR was not determinable for fulvestrant because there was only one objective response.

“Today’s approval of Revtorpyk addresses a significant unmet need for the thousands of patients affected each year by HR+/HER2-, PIK3CA wild-type locally advanced or metastatic breast cancer whose disease has progressed after endocrine therapy,” said Igor Gorbatchevsky, MD, Chief Medical Officer of Celcuity. “We are deeply grateful to the patients and their caregivers, investigators and clinical study teams, and Celcuity team members who made this advancement possible.”

Celcuity anticipates commercial launch in late Q3 2026. Based on the company’s commitment to ensuring broad, affordable, and unrestricted patient access to Revtorpyk, we have designed a comprehensive patient support program. To make Revtorpyk available to patients prior to commercial launch, those who are eligible will be able to enroll in Celcuity’s expanded access program.

Celcuity plans to submit in Q3 2026 a supplemental New Drug Application (“sNDA”) to the FDA for Revtorpyk for the treatment of HR+/HER2-, PIK3CA mutated, locally advanced or metastatic breast cancer, following at least one line of endocrine therapy based on results from the mutant cohort of the Phase 3 VIKTORIA-1 trial. Results for this study were recently presented in a late-breaking abstract oral session at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting. Following the sNDA submission, Celcuity intends to submit VIKTORIA-1 data for marketing authorization of gedatolisib to other regulatory authorities around the world.

Revtorpyk is also being studied in the ongoing Phase 3 VIKTORIA-2 clinical trial incorporating two independent studies evaluating two separate patient cohorts with HR+/HER2- locally advanced or metastatic breast cancer who are treatment-naive in the advanced setting.

About Revtorpyk (gedatolisib)

Revtorpyk is a kinase inhibitor of class I PI3K isoforms (α, β, δ, γ) and mTOR complexes mTORC1 and mTORC2, resulting in downstream inhibition of multiple effectors, including AKT.3.4.5

Revtorpyk is in development for the first-line treatment of HR+/HER2- locally advanced or metastatic breast cancer and for the second-line treatment of metastatic castration resistant prostate cancer.

Indication Statement

Revtorpyk (gedatolisib) is a kinase inhibitor indicated in combination with fulvestrant, with or without palbociclib, for the treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after treatment with at least one line of endocrine therapy in the metastatic setting.

https://en.wikipedia.org/wiki/Gedatolisib

FDA Approves Revtorpyk (gedatolisib) for the Treatment of HR+/HER2-, PIK3CA Wild-Type Locally Advanced or Metastatic Breast Cancer

FDA Accepts New Drug Application for Genentech’s Giredestrant in ESR1-Mutated, ER-Positive Advanced Breast Cancer

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced  the U.S. Food and Drug Administration (FDA)  acceptance of  the company’s New Drug Application for giredestrant, an investigational oral therapy, in combination with everolimus for the treatment of adult patients with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated locally advanced or metastatic breast cancer following recurrence or progression on a prior endocrine-based regimen. The FDA is expected to make a decision on the approval by December 18, 2026. Giredestrant plus everolimus could be the first and only oral selective estrogen receptor degrader (SERD) combination approved in the post-cyclin-dependent kinase (CDK)4/6 inhibitor setting.

Filing acceptance based on Phase III data showing giredestrant plus everolimus reduced the risk of disease progression or death by 44% and 62% in ITT and ESR1-mutated populations, respectively, versus standard-of-care endocrine therapy plus everolimus

Strength of evERA data demonstrate potential for giredestrant combination to help address resistance to standard-of-care therapies, and could be the first and only oral SERD combination approved in the post-CDK4/6 inhibitor setting

“The clinically meaningful benefit seen with giredestrant could enable an important new treatment option to help delay disease progression or death in people with advanced, ER-positive breast cancer,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “This acceptance marks a first step towards establishing the giredestrant combination as a new standard of care in this population.”

The filing acceptance is based on the Phase III evERA Breast Cancer study results, which showed that giredestrant plus everolimus reduced the risk of disease progression or death by 44% and 62% in the intention-to-treat (ITT) and ESR1-mutated populations, respectively, compared with standard-of-care endocrine therapy plus everolimus. In the ESR1-mutated population, the median progression-free survival (PFS) was 9.99 months compared with 5.45 months in the giredestrant and comparator arm, respectively (stratified hazard ratio [HR]=0.38, 95% CI: 0.27-0.54, p-value=<0.0001). In the ITT population, the median PFS was 8.77 months compared with 5.49 months in the giredestrant and comparator arms, respectively (HR=0.56, 95% CI: 0.44-0.71, p-value=<0.0001).

Overall survival (OS) data were immature at the time of analysis, but a clear positive trend has been observed in the ITT (HR=0.69, 95% CI: 0.47-1.00, p-value=0.0473) and ESR1-mutated populations (HR=0.62, 95% CI: 0.38-1.02, p-value=0.0566). Follow-up for OS will continue to the next analysis. Adverse events for the giredestrant combination were manageable and consistent with the known safety profiles of the individual medicines. No unexpected safety findings were observed, including no photopsia.

Data from evERA are being used to support filing submissions to other global health authorities.

ER-positive breast cancer accounts for approximately 70% of breast cancer cases. Resistance to endocrine therapies, particularly in the post-CDK inhibitor setting, increases the risk of disease progression and is associated with poor outcomes. Oral combination therapies, such as giredestrant plus everolimus, could address this by targeting two different signaling pathways while helping to minimize the impact of treatment on people’s lives without the need for injections.

evERA was the first positive Phase III readout for giredestrant, followed by lidERA Breast Cancer in the early-stage setting. The scientific rationale for lidERA was supported by prior results in the neoadjuvant setting, including the coopERA trial showing that giredestrant was superior to an aromatase inhibitor in reducing malignant cell division (Ki67 levels). This growing body of evidence underscores the potential of giredestrant to become a new standard-of-care endocrine therapy across ER-positive early-stage and advanced breast cancer. In the coming weeks, Genentech will submit the giredestrant Phase III lidERA data in early-stage breast cancer to the FDA. The persevERA readout in first-line ER-positive breast cancer is expected in the first half of this year, which will provide further evidence for giredestrant in the ER-positive breast cancer treatment paradigm.

Our extensive giredestrant clinical development program spans multiple treatment settings and lines of therapy, reflecting our commitment to deliver innovative medicines to as many people with ER-positive breast cancer as possible.

https://en.wikipedia.org/wiki/Giredestrant

Xspray Pharma Re-Submits its FDA Application for Dasynoc

Xspray Pharma AB (publ) has re-submitted its application for market approval for Dasynoc® to the U.S. Food and Drug Administration (FDA). Dasynoc® is the company's lead product candidate – an amorphous dasatinib for the treatment of leukemia. The resubmission includes the additional information requested by the FDA. A new PDUFA-date is expected to be set for August 2026 at the latest.

Xspray Pharma has submitted its response to the CRL (Complete Response Letter) received from the FDA in October 2025. The CRL related to the company’s NDA application (New Drug Application) for market approval in the United States for Dasynoc, an amorphous dasatinib for the treatment of CML (chronic myeloid leukemia) and ALL (acute lymphoblastic leukemia).

The current resubmission includes additional data specifically designed to address FDA’s concerns regarding risk for medication error. A qualitative human factors study has been completed to address this, confirming high prescriber comprehension using the new tablet strengths. The requested documentation confirming the effectiveness of previously implemented manufacturing measures was also submitted.

The GMP-observations in the company’s third-party manufacturing facility are being handled by the facility itself, and corrective measures are being implemented. Xspray welcomes the recent acquisition of the facility by Benta Group, a well-established company with core operations in pharmaceutical contract manufacturing and a strong reputation for quality, regulatory compliance and operational standards.

“Although the progress and approval process of the facility is not under our direct control, current information that is available to us reassures us that the issues in the facility will be resolved. We maintain our plan to launch two products, Dasynoc and XS003 nilotinib during the second half of 2026,” comments Per Andersson, CEO of Xspray Pharma.

The Dasynoc application will now undergo FDA review. Once the FDA formally confirms acceptance of the application, a new PDUFA date will be announced, expected no later than August 2026.

https://en.wikipedia.org/wiki/Dasatinib

Telix Resubmits NDA to U.S. FDA for TLX101-Px (Pixclara) Brain Cancer Imaging Candidate

Telix Pharmaceuticals Limited (ASX: TLX, NASDAQ: TLX, “Telix”) announced the resubmission of a New Drug Application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for TLX101-Px, (Pixclara®1, Floretyrosine F 18 or 18F-FET), an investigational PET2 imaging agent for the characterization of recurrent or progressive glioma (brain cancer) from treatment related changes in both adult and pediatric patients.

Telix has resubmitted the NDA with the additional data requested by the FDA. The Company believes, based on the Type A meeting and ongoing consultation with the FDA, that the additional data and statistical analysis, along with the primary data set provided in the original submission, appropriately addresses the Complete Response Letter3.

Given the potential to address significant unmet medical need, TLX101-Px has been granted Orphan Drug4 and Fast Track5 designations by the FDA. PET imaging with 18F-FET is already included in international clinical practice guidelines for the imaging of gliomas6, however there is currently no FDA-approved targeted amino acid PET agent for adult and pediatric brain cancer imaging commercially available in the U.S.

Dr. David N. Cade, Telix Group Chief Medical Officer, said, “We appreciate the FDA’s recognition of the critical unmet need to improve the diagnosis and management of glioma, particularly in the post-treatment setting. Our resubmission is supported by an extensive and compelling data set – particularly so for an orphan indication. We are grateful to our global clinical collaborators, who share our commitment to ensuring patients in the U.S. can benefit from this important patient management tool.”

Maggie Haynes, Executive Director, Head for the Cure Foundation, added: “Our community is encouraged by the FDA’s ongoing engagement and guidance to the sponsor and support for the Expanded Access Program for TLX101-Px. We are hopeful of an expedited review, so this important and proven imaging option can become available to those who urgently need it.”

https://en.wikipedia.org/wiki/Fluoroethyl-L-tyrosine_(18F)

Nuvalent Announces Submission of New Drug Application to FDA for Neladalkib in TKI Pre-treated Advanced ALK-Positive NSCLC

Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced the submission to the U.S. Food and Drug Administration (FDA) of the Company's NDA for neladalkib, an investigational ALK-selective inhibitor, in TKI pre-treated advanced ALK-positive NSCLC.

New Drug Application (NDA) based on data in TKI pre-treated patients from the global ALKOVE-1 Phase 1/2 clinical trial

"The advancement of neladalkib from first clinical trial initiation to NDA submission in less than four years represents a remarkable pace in oncology drug development, underscoring the vigor and urgency our team brought to this program and our deep commitment to the ALK-positive NSCLC community," said Darlene Noci, A.L.M., Chief Development Officer at Nuvalent. "We would like to extend our sincere gratitude to the patients, families and investigators who have made this progress possible, and are committed to working closely with the FDA throughout the NDA review process toward our goal of bringing neladalkib to patients as quickly as possible."

The application is based on data in TKI pre-treated patients with advanced ALK-positive NSCLC treated with neladalkib in the global, registration-directed ALKOVE-1 Phase 1/2 clinical trial. In this population, neladalkib demonstrated encouraging overall activity, including intracranial responses, the ability to address key drivers of disease progression, and a generally well-tolerated safety profile consistent with its ALK-selective, TRK-sparing design. The company plans to share detailed results at a future medical meeting.

Neladalkib has received breakthrough therapy designation from the FDA for the treatment of patients with locally advanced or metastatic ALK-positive NSCLC who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

About Neladalkib

Neladalkib is an investigational, brain-penetrant, ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

https://go.drugbank.com/drugs/DB16688